Journal of neuromuscular diseases
Meeting report: 2024 Muscular Dystrophy Association summit on 'Safety and challenges in gene therapy of neuromuscular diseases'.
Angela Lek, Evrim Atas, Brian Lin, Sharon E Hesterlee, Carsten G Bönnemann, Barry J Byrne
Published: 202610.1177/22143602251364325
Abstract
This meeting report summarizes the presentations and discussions held at the summit on 'Challenges in Gene Therapy' hosted by the Muscular Dystrophy Association (MDA) in 2024. Topics broadly cover in vitro and in vivo models for understanding of gene…
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